Rare genetic variants raise the risk of ADHD
A significant share of the risk and heritability of attention-deficit hyperactivity disorder (ADHD) is explained by rare genetic variants. A study led by scientists from Aarhus University in Denmark has uncovered their weight in this condition and identified three variants that will help to better understand their role, the risk of developing it, or its comorbidities, in contrast with the common and more frequent variants associated with ADHD.

Rare genetic variants raise the risk of ADHD
A significant share of the risk and heritability of attention-deficit hyperactivity disorder (ADHD) is explained by rare genetic variants. A study led by scientists from Aarhus University in Denmark has uncovered their weight in this condition and identified three variants that will help to better understand their role, the risk of developing it, or its comorbidities, in contrast with the common and more frequent variants associated with ADHD.
UK Biobank delivers largest metabolomics dataset of 500K profiles
It’s the biological resource that keeps on giving, and now UK Biobank has released the final tranche of data on the levels of 249 metabolites in the blood of its half a million participants.
Evaxion reports antigen data from EVX-V1 CMV vaccine program
Evaxion A/S is advancing its cytomegalovirus (CMV) vaccine program, EVX-V1, with new data demonstrating protective effects of lead antigens in the program. EVX-V1 is a next-generation, multicomponent vaccine program combining novel AI-discovered antigens with AI-optimized versions of established CMV vaccine antigens.
Revir reports milestones in HTT-PMS1 Huntington’s disease program
Revir Therapeutics Inc. has announced progress in its HTT-PMS1 genetic medicine program for Huntington’s disease with the identification of a lead compound and the award of a $4.6 million grant from the California Institute for Regenerative Medicine (CIRM).
Eikonizo’s HDAC6 inhibitor improves HFpEF pathology
Heart failure with preserved ejection fraction (HFpEF) is a complex disease with limited therapeutic options. Protein histone deacetylase 6 (HDAC6) is known to be involved in several biological processes, such as autophagy or inflammation, among others, but its impact on HFpEF has not been well evaluated to date.
Celebrating BioWorld's 35th anniversary
Managing Editor Anette Breindl talks about covering research breakthroughs that later became industry defining.
TUB-030 shows potent activity in 5T4-positive sarcoma
Researchers at Tubulis GmbH reported preclinical efficacy data on TUB-030, a novel 5T4-targeting antibody-drug conjugate (ADC), in several sarcoma subtypes.
Humanwell Healthcare patents new MRGPRX4 antagonists
Humanwell Healthcare (Group) Co. Ltd. has disclosed Mas-related G-protein coupled receptor member X4 (MRGPRX4; SNSR5; SNSR6) antagonists reported to be useful for the treatment of pruritus.
IPT-001 exerts tumor regression through HERV-K targeting
Human endogenous retroviruses (HERVs) are residues from ancient viral infections that have become integrated into the human genome. One of them – HERV-K – is aberrantly reactivated in solid tumors but silent in healthy tissues.
Novel B7-H7-targeted ADC NPX-125 active in solid tumors
Nextpoint Therapeutics Inc. presented their novel B7-H7-directed antibody-drug conjugate (ADC).

Conferences

EP-102 METTL3 inhibitor shows efficacy in NSCLC models
AACR-NCI-EORTC Molecular Targets and Cancer Therapeutics
METTL3, the enzyme that adds the m6A RNA modification, is a key regulator of RNA processing and protein synthesis. In cancer, METTL3 is often overexpressed, driving tumor growth, invasion and therapy resistance across multiple malignancies, including lung, pancreatic, ovarian, colorectal cancers...
Pilatus’ PLT-012 exerts tumor control in liver cancer
Society for Immunotherapy of Cancer
Pilatus Biosciences SA has developed a CD36-targeting humanized antibody, PLT-012, for the potential treatment of cancer.
NS-136 is a safe and effective M4 PAM antipsychotic
Neurology/psychiatric
Schizophrenia is a complex psychiatric disorder marked by positive, negative and cognitive symptoms, many of which are poorly addressed by current dopamine-blocking antipsychotics. Preclinical studies show that muscarinic acetylcholine M4 receptor positive allosteric modulators (M4 PAMs) can reduce...
Modex’s MDX-2004 enhances antitumor immunity, study shows
Cancer
The limited efficacy of cancer immunotherapy is usually attributed to suboptimal antitumor T-cell generation, as well as impaired immunological memory development. MDX-2004 is a trispecific antibody-fusion protein developed by Modex Therapeutics Inc. that targets CD3, CD28 and CD137 on human T...

Cancer

Antengene outlines early-stage pipeline
Immune
Antengene Corp. Ltd. has outlined progress in its early-stage pipeline. The company has leveraged its Antengager T-cell engager (TCE) platform for the discovery of multiple investigational programs.

Infection

Mabloc and Instituto Butantan partner on MBL-YFV-01
Collaboration
Mabloc LLC has established a strategic partnership with Instituto Butantan to co-develop and manufacture MBL-YFV-01, a monoclonal antibody therapy for yellow fever virus (YFV) infection, including emergency post-exposure use.

‘Most complete’ map of oral microbiome enables links to systemic disease
Artificial intelligence
South Korean researchers led by Lee In-suk of Yonsei University have reported the most complete oral microbiome catalog to date, with more than 72,000 genomes. Detailed in Cell Host & Microbe on Nov. 12, 2025, the database is expected to serve as a universal platform for academia and enable...
Scynexis fungerps among funding recipients
Scynexis Inc. has announced that a novel series of antifungal compounds utilizing its proprietary triterpenoid antifungal platform are among five projects funded by a federal grant awarded to the new accelerator consortium led by researchers from Hackensack Meridian Center for Discovery and...

Neurology/psychiatric

Ventoux’s VEN-201 to be studied for spinal cord injury
Ventoux Biosciences Inc. has initiated a research program to explore the therapeutic potential of lead compound, VEN-201, in translational preclinical models of spinal cord injury.
Aperture Therapeutics nominates APRTX-001 as candidate
Antisense
Aperture Therapeutics Inc. has nominated APRTX-001 as a development candidate, with the program now advancing through IND-enabling studies. APRTX-001 is a CD33-targeting antisense oligonucleotide (ASO) designed for the treatment of frontotemporal dementia and amyotrophic lateral sclerosis, with...
Multiomic study shows antiaging effects of GLP-1RAs
Science
Could GLP-1 receptor agonists (GLP-1RAs), already used in obesity and diabetes, be repurposed as drugs to slow aging? Hong Kong, one of the places in the world with the highest human longevity, is also home to a scientific study on the effects of GLP-1RAs. For the first time, scientists at the...
SfN 2025: How ‘latent’ sex differences can trip up drug discovery
Science
At the 2025 annual meeting of the Society for Neuroscience in San Diego this week, Catherine Woolley’s plenary lecture was an unusual combination of debunking and affirming the importance of sex differences in the brain.
Idorsia Pharmaceuticals describes new OX2 receptor agonists
Patents
Idorsia Pharmaceuticals Ltd. has identified aryl sulfone and sulfanone derivatives acting as orexin OX2 receptor (HCRTR2) agonists reported to be useful for the treatment of eating disorders, fatigue, Kleine-Levin syndrome, narcolepsy, obesity, pain, and psychiatric and inflammatory disorders,...

Endocrine/metabolic

Mitorx Therapeutics raises funds to advance Myo-004 for obesity
Financings
Mitorx Therapeutics Ltd. has raised £5.5 million (US$7.2 million) in a pre-series A round to advance its lead mitochondrial-targeted program, Myo-004, through clinical candidate selection.

Biomarkers

With transdiagnostic approach, smaller target groups aren’t inevitably smaller markets
Neurology/psychiatric
Despite the formidable challenges for developing precision psychiatry, the approach is notching its first successes in the preclinical and even some clinical settings. Many individual studies as well as large projects like the Psychiatric Ratings using Intermediate Markers (PRISM) studies and the...

Anchoring, and bootstrapping, psychiatry into the precision medicine era
Neurology/psychiatric
Psychiatry has struggled to enter the precision medicine era. But through a mix of innovations and bootstrapping, progress is coming to the field. Scientists are working on improving diagnoses by investigating potential biomarkers and collection methods.
Biomarker panel of three serum microRNAs predicts cervical cancer prognosis
Cancer
MicroRNAs (miRNAs) play key roles in cancer development by regulating genes involved in cell growth, differentiation, invasion, metastasis and angiogenesis. Because their expression patterns differ across tumor types and stages, miRNA profiles hold strong potential as noninvasive diagnostic and...

Gastrointestinal

UK study reveals inconsistencies in global microbiome research
Regulatory
The U.K. Medicines and Healthcare products Regulatory Agency is calling for unified standards to harmonize microbiome research, after revealing major inconsistencies in the results when labs around the world analyzed identical reference samples of...
Samjin Pharmaceutical patents new compounds for MASH
Patents
Samjin Pharmaceutical Co. Ltd. has disclosed inhibitors of 17-β-hydroxysteroid dehydrogenase 13 (HSD17B13; 17-β-HSD 13) and/or estradiol 17-β-dehydrogenase 1 (HSD17B1; 17β-HSD1) and/or HSD17B2 (17β-HSD2). As such, they are believed to be...
Imhotex patents new NOD2 activators for Crohn’s disease
Patents
Imhotex Ltd. has disclosed desmuramylpeptide (DMP) analogues of muramyl dipeptide (MDP) acting as nucleotide-binding oligomerization domain-containing protein 2 (NOD2) activators reported to be useful for the treatment of Crohn’s disease.
Numab and Kaken sign agreement for NM-81 for IBD
Collaboration
Numab Therapeutics AG and Kaken Pharmaceutical Co. Ltd. have entered into a strategic licensing and co-development agreement for NM-81, a multi-specific antibody for the treatment of inflammatory bowel disease (IBD).
Korro Bio nominates KRRO-121 as development candidate
Endocrine/metabolic
Korro Bio Inc. has nominated KRRO-121 as its next development candidate, for the treatment of patients with hyperammonemia, including patients with urea cycle disorders and hepatic encephalopathy.
Genfit and Everzom form research collaboration in liver failure
Collaboration
Genfit SA has established a research collaboration with Everzom SAS to expand its acute-on-chronic liver failure (ACLF) research via exosome-based regenerative technology. The partners plan to conduct exploratory studies to assess efficacy of...

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